Ziftomenib

Kura Oncology and Kyowa Kirin collaborate globally to develop and commercialize ziftomenib, a selective oral menin inhibitor for AML and other hematologic malignancies. Kura receives $330M upfront and up to $1.161B in milestone payments. Kura leads US development and commercialization, while Kyowa Kirin handles global commercialization, with Kura eligible for royalties. Ziftomenib, with FDA breakthrough designation for R/R NPM1-mutant AML, aims for NDA submission in 2025.

Kura Oncology secures $330 million from Kyowa Kirin for ziftomenib, a menin inhibitor targeting KMT2A-mutated leukemia, with plans for FDA submission in 2025. The collaboration aims to develop ziftomenib across leukemias and solid tumors, with potential milestone payments up to $1.2 billion.

Kura Oncology Inc. and Kyowa Kirin Co., Ltd. form global partnership to develop and commercialize ziftomenib, a selective oral menin inhibitor for treating AML and other blood cancers. Kura receives $330 million upfront and anticipates up to $1.161 billion in milestone payments. Kura leads U.S. development and commercial strategy, while Kyowa Kirin handles global commercialization, with Kura eligible for tiered royalties. Ziftomenib is the first therapy to receive FDA breakthrough designation for R/R NPM1-mutant AML, with an anticipated NDA submission in 2025.

Syndax Pharmaceuticals' drug Revuforj, a menin inhibitor, received FDA approval for treating acute leukemia with KMT2A translocation, offering a new treatment option for patients with poor prognoses. The drug, priced at $39,500/month, showed promising results in a Phase 1/2 study, with 21.2% achieving complete remission. Revuforj carries a black box warning for differentiation syndrome risk.

FDA approves Syndax Pharma's Revuforj, an oral menin inhibitor, for relapsed or refractory acute leukaemia with KMT2A gene translocations, marking the first FDA-backed treatment for this type of blood cancer. This follows a disappointing phase 1/2 trial readout for Revuforj in mutant NPM1 acute myeloid leukaemia, causing a drop in Syndax's shares.

Syndax Pharmaceuticals' Phase II trial of revumenib met primary endpoint in relapsed or refractory AML, but stock dropped 25.6%. The AUGMENT-101 trial showed 23% CR/CRh, 4.7 months median duration, and 64% MRD-negative patients. Safety issues included QTc prolongation and differentiation syndrome. Revumenib targets menin-MLL interaction, competing with Kura Oncology's ziftomenib. Revumenib projected to generate $634m by 2030.

Kura Oncology reports Q3 2024 financial results, expecting topline results from ziftomenib trial in R/R NPM1-mutant AML in early 2025. The company plans to present data from 100 patients in the Phase 1a study at ASH, with the Phase 1b expansion study enrolling at 600 mg. Preclinical data supports ziftomenib's potential in GIST, with a proof-of-concept study expected in 1H 2025. Kura also dosed the first patient in the KO-2806 and adagrasib study in KRASG12C-mutated NSCLC. The company reported a net loss of $54.4 million for Q3 2024, with R&D expenses at $41.7 million and G&A expenses at $18.2 million. Kura holds $455.3 million in cash, cash equivalents, and investments, expected to fund operations into 2027.

Kura Oncology to report Q3 2024 financial results on Nov 7, 2024, with a webcast and conference call at 4:30 p.m. ET / 1:30 p.m. PT. The call can be accessed via phone or webcast on the company's website.

Stifel downgraded Kura Oncology's stock from Buy to Hold, reducing the price target to $19.00 due to anticipated challenges for ziftomenib in the cancer drug market. Despite progress with ziftomenib, including a breakthrough therapy designation and FDA clearance, Kura reported a net loss of $50.8 million. The company's strong cash position of $491.5 million is expected to fund operations into 2027, and recent advancements include the addition of Dr. Michael J. Vasconcelles to the Board of Directors.

The KOMET-001 study, led by Roswell Park, tested ziftomenib, a menin inhibitor, in AML patients with prior therapies. Published in The Lancet Oncology, it showed partial or complete response in about a third of patients, particularly those with NPM1 mutations or KMT2A rearrangements. The FDA granted Breakthrough Therapy designation to ziftomenib, and additional trials are underway at Roswell Park.